Trial Outcomes & Findings for Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy (NCT NCT02173158)
NCT ID: NCT02173158
Last Updated: 2018-10-10
Results Overview
Mean percent change from baseline
COMPLETED
PHASE3
9 participants
Baseline to Week 26
2018-10-10
Participant Flow
Participant milestones
| Measure |
Lomitapide
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Efficacy Phase
STARTED
|
9
|
|
Efficacy Phase
COMPLETED
|
8
|
|
Efficacy Phase
NOT COMPLETED
|
1
|
|
Safety Phase
STARTED
|
8
|
|
Safety Phase
COMPLETED
|
8
|
|
Safety Phase
NOT COMPLETED
|
0
|
Reasons for withdrawal
| Measure |
Lomitapide
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Efficacy Phase
Adverse Event
|
1
|
Baseline Characteristics
Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
Baseline characteristics by cohort
| Measure |
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Age, Continuous
|
50.3 years
STANDARD_DEVIATION 14.71 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 26Population: Full analysis set for the efficacy phase, included all subjects who received study drug and had a baseline and at least one post-baseline assessment
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Percent Change in LDL-C
|
-42.2 Percent change
Standard Deviation 18.16
|
SECONDARY outcome
Timeframe: Baseline to Week 56Population: Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=8 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Change in Total Cholesterol
|
-25.9 Percent change
Standard Deviation 18.57
|
SECONDARY outcome
Timeframe: Baseline to Week 56Population: Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=8 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Change in Apo B
|
-41.4 Percent change
Standard Deviation 21.90
|
SECONDARY outcome
Timeframe: Baseline to Week 56Population: Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=8 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Change in Triglycerides
|
-44.4 Percent change
Standard Deviation 12.70
|
SECONDARY outcome
Timeframe: Baseline to Week 56Population: Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=8 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Change in Non-HDL-C
|
-34.6 Percent change
Standard Deviation 22.50
|
SECONDARY outcome
Timeframe: Baseline to Week 56Population: Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=8 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Change in VLDL-C
|
-44.8 Percent change
Standard Deviation 12.05
|
SECONDARY outcome
Timeframe: Baseline to Week 56Population: Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=8 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Change in Lp(a)
|
-27.2 Percent change
Standard Deviation 23.50
|
SECONDARY outcome
Timeframe: Baseline to Week 56Population: Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=8 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Change in HDL-C
|
5.9 Percent change
Standard Deviation 19.64
|
SECONDARY outcome
Timeframe: Baseline to Week 56Population: Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=8 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Change in Apo AI
|
-2.8 Percent change
Standard Deviation 11.61
|
SECONDARY outcome
Timeframe: Baseline to Week 56Population: Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase
Mean percent change from baseline
Outcome measures
| Measure |
Lomitapide
n=8 Participants
Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
|
|---|---|
|
Change in LDL-C
|
-37.5 Percent change
Standard Deviation 24.21
|
Adverse Events
Efficacy Phase
Safety Phase
Serious adverse events
| Measure |
Efficacy Phase
n=9 participants at risk
Baseline to Week 26
|
Safety Phase
n=8 participants at risk
Week 26 to Week 56
|
|---|---|---|
|
General disorders
Chest pain
|
0.00%
0/9
|
12.5%
1/8 • Number of events 1
|
Other adverse events
| Measure |
Efficacy Phase
n=9 participants at risk
Baseline to Week 26
|
Safety Phase
n=8 participants at risk
Week 26 to Week 56
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
77.8%
7/9
|
50.0%
4/8
|
|
Gastrointestinal disorders
Nausea
|
22.2%
2/9
|
12.5%
1/8
|
|
Gastrointestinal disorders
Abdominal distension
|
11.1%
1/9
|
0.00%
0/8
|
|
Gastrointestinal disorders
Abdominal pain lower
|
11.1%
1/9
|
0.00%
0/8
|
|
Gastrointestinal disorders
Dental caries
|
11.1%
1/9
|
0.00%
0/8
|
|
Gastrointestinal disorders
Faeces soft
|
11.1%
1/9
|
0.00%
0/8
|
|
Gastrointestinal disorders
Flatulence
|
11.1%
1/9
|
0.00%
0/8
|
|
Gastrointestinal disorders
Haemorrhoids
|
11.1%
1/9
|
12.5%
1/8
|
|
Infections and infestations
Nasopharyngitis
|
44.4%
4/9
|
37.5%
3/8
|
|
Infections and infestations
Gastroenteritis
|
22.2%
2/9
|
12.5%
1/8
|
|
Infections and infestations
Influenza
|
11.1%
1/9
|
12.5%
1/8
|
|
Infections and infestations
Periodontitis
|
11.1%
1/9
|
12.5%
1/8
|
|
Infections and infestations
Pneumonia
|
11.1%
1/9
|
0.00%
0/8
|
|
General disorders
Chest pain
|
0.00%
0/9
|
12.5%
1/8
|
|
General disorders
Device breakage
|
0.00%
0/9
|
12.5%
1/8
|
|
General disorders
Feeling abnormal
|
11.1%
1/9
|
12.5%
1/8
|
|
General disorders
Malaise
|
11.1%
1/9
|
0.00%
0/8
|
|
Injury, poisoning and procedural complications
Chemical burn of skin
|
0.00%
0/9
|
12.5%
1/8
|
|
Injury, poisoning and procedural complications
Heat stroke
|
0.00%
0/9
|
12.5%
1/8
|
|
Injury, poisoning and procedural complications
Contusion
|
11.1%
1/9
|
0.00%
0/8
|
|
Injury, poisoning and procedural complications
Subcutaneous haematoma
|
11.1%
1/9
|
0.00%
0/8
|
|
Injury, poisoning and procedural complications
Tooth fracture
|
11.1%
1/9
|
0.00%
0/8
|
|
Blood and lymphatic system disorders
Anaemia
|
22.2%
2/9
|
25.0%
2/8
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
0.00%
0/9
|
12.5%
1/8
|
|
Investigations
Liver function test abnormal
|
33.3%
3/9
|
12.5%
1/8
|
|
Investigations
Blood creatine phosphokinase increased
|
11.1%
1/9
|
12.5%
1/8
|
|
Investigations
White blood cell count decreased
|
0.00%
0/9
|
12.5%
1/8
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
11.1%
1/9
|
0.00%
0/8
|
|
Musculoskeletal and connective tissue disorders
Mylagia
|
11.1%
1/9
|
0.00%
0/8
|
|
Nervous system disorders
Headache
|
22.2%
2/9
|
0.00%
0/8
|
|
Skin and subcutaneous tissue disorders
Drug eruption
|
11.1%
1/9
|
0.00%
0/8
|
|
Skin and subcutaneous tissue disorders
Eczema
|
11.1%
1/9
|
0.00%
0/8
|
|
Skin and subcutaneous tissue disorders
Pruritus allergic
|
11.1%
1/9
|
0.00%
0/8
|
|
Skin and subcutaneous tissue disorders
Rash
|
11.1%
1/9
|
12.5%
1/8
|
|
Cardiac disorders
Palpitations
|
11.1%
1/9
|
0.00%
0/8
|
|
Hepatobiliary disorders
Hepatic function abnormal
|
11.1%
1/9
|
12.5%
1/8
|
|
Metabolism and nutrition disorders
Decreased appetite
|
11.1%
1/9
|
0.00%
0/8
|
Additional Information
Alison Long, MD - VP Clinical
Aegerion Pharmaceuticals, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee Described in site contract
- Publication restrictions are in place
Restriction type: OTHER